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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-001628-16 | EudraCT Number |
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This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed dose combination (FDC) tablet for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12 | Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM). | Baseline, Week 8 through Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12 | Baseline, Week 8 through Week 12 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royal Adelaide Hospital | Adelaide | Australia | ||||
| The Prince Charles Hospital |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
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This study was conducted in participants with cystic fibrosis, aged 12 years and older, who are heterozygous for the F508del mutation and the minimal function mutation (F/MF) genotypes.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/ tezacaftor (TEZ) 100 mg qd/ ivacator (IVA) 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 30, 2021 | Jun 12, 2023 |
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| IVA | Drug | Tablet for oral administration. |
|
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| Chermside |
| Australia |
| Alfred Hospital | Melbourne, VIC | Australia |
| Institute for Respiratory Health | Nedlands | Australia |
| Telethon Kids Institute | Nedlands | Australia |
| The Royal Children's Hospital | Parkville, VIC | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| University of Calgary Medical Clinic of the Foothills Medical Centre | Calgary | Canada |
| University of Alberta Hospital, Edmonton Clinic | Edmonton | Canada |
| Queen Elizabeth II Health Sciences Center | Halifax | Canada |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | Spain |
| Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari | Sabadell | Spain |
| COMPLETED |
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| NOT COMPLETED |
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All participants who received at least one dose of the study drug during the treatment period were included in the baseline analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period approximately 13 weeks. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12 | Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM). | The Full Analysis Set (FAS) will include all enrolled participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome. | Posted | Least Squares Mean | 95% Confidence Interval | percent reduction | Baseline, Week 8 through Week 12 |
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| Secondary | Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12 | FAS. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome. | Posted | Least Squares Mean | 95% Confidence Interval | step count per day | Baseline, Week 8 through Week 12 |
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Day 1 up to Week 17
Safety Set included all participants who had received at least 1 dose of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ELX/TEZ/IVA | Participants received ELX 200 mg qd/ TEZ 100 mg qd/ IVA150 mg q12h in the treatment period approximately 13 weeks. | 0 | 81 | 2 | 81 | 56 | 81 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pancreatitis | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 24, 2022 | Jun 12, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Title | Denominators | Categories | ||||
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